Chinese scientists have developed CS-101, the world's first gene therapy for thalassemia, enabling patients to become independent of blood transfusions.

Foreign Media: Chinese scientists have developed CS-101, the world's first gene therapy for thalassemia, bringing hope for a cure to this most common inherited blood disorder. The therapy, developed by scientists at ShanghaiTech University using tBE base editing technology, combines gene editing with stem cell technology.

Five beta-thalassemia patients who received the treatment rapidly regained hematopoietic function, with total hemoglobin and fetal hemoglobin levels rising quickly and remaining elevated, allowing them to become independent of blood transfusions.

Globally, there are 350 million carriers of thalassemia. China currently has 30 million gene carriers, and severe patients require lifelong blood transfusions, facing risks of premature death; if two carriers have children, there is a 25% chance their offspring will be affected.

Original article: toutiao.com/article/1863725363602504/

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